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TRANSCRIPT
Thank you for joining ISMPP U today!
The program will begin
promptly at 11:00 am EST
ISMPP would like to thank…
the following Platinum Sponsors for their ongoing support of the society
Today’s Program
• Presenter Chris L. Pashos, PhD
• Moderator Michael Wittek, MSW
• Q&A follows the presentation−but feel free to send in questions as you have them
HEOR 201
How Valuable is this Medical Technology?The Role of Health Economics and Outcomes Research
Chris L. Pashos, PhD
November 18, 2009
ISMPP U
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Outline: The Role of Health Economics &
Outcomes Research
I. Linking Clinical Trials to the Real World
II. Understanding the Burden of Disease
III. Projecting the Value of Interventions
IV. Providing Real-World Proof
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I. Linking Clinical Trials to the
Real World
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Marketplace Evolution
• The challenges to achieve market success have changed over time
– Before the 1980s, regulatory approval assured commercial success of
safe and efficacious products
– In the 1980s and 1990s, regulatory approval with some economic and
Patient Reported Outcomes data to support reimbursement assured
commercial viability
• The present and future
– Regulatory approval with data in controlled settings gets sponsors to
the starting line
– To achieve commercial success, sponsors need to marshal resources
efficiently and effectively to plan and implement an integrated market
access, marketing and sales effort
– Sponsors must dramatically improve their ability to produce scientifically
valid real-world evidence to demonstrate the value of their products
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• “… we need a new approach to analysing clinical
evidence”
• “… RCTs’ appearance at the top of the heirarchy of
evidence is inappropriate… need a diversity of approaches
that involve the totality of the evidence base”
• “…observational studies are also useful… can provide an
important source of evidence about both the benefits and
harms of therapeutic interventions”
Sir Michael Rawlins’ statements in October 2008 to the
Royal College of Physicians
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• In October 2009, the AMCP released the
latest version of its dossier submission
guidelines.
•The one substantive change from format v2.1
is the requirement to include published data
from retrospective observational studies,
meta-analyses, assessments of adherence or
persistence, studies of patient preference, and
other HEOR analyses.
AMCP Format for Formulary Submissions, Version 3.0
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Context: Key Stakeholders
• Clinicians
• Physician and disease organizations
• Health technology industry
• Health technology assessment agencies
• Health insurers / payers
• Regulatory authorities
• Patients
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• Involves the collection and analysis of data on the
use of health care products and services and on their
clinical, economic, and patient-reported outcomes.
• Synthesizes both real-world and clinical trial data to
provide the sponsor and key stakeholders -
physicians, health care providers, regulators, payers
and patients – via publications, a comprehensive
view of the evidence on the burden of disease and
the value of specific products in reducing that burden.
Health Economics and Outcomes Research
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Health Economics and Outcomes Research Involves
and Integrates Tools from Multiple Disciplines
RetrospectiveObservational
Study
PatientRegistry
Market Assessment
ExploratoryData
Assessment
Meta-AnalysisAssessment of
Compliance andPersistence
Budget Impact
Modeling
Sales and Revenue
Forecasting
Pricing Strategy
Cost of IllnessAnalysis
SatisfactionAssessment
Reimbursement Analysis
Payor Assessment
Scenario Testing
Biometrics
Competitive Analysis
HRQL Analysis
Risk Assessment
Cost-Effectiveness
Modeling Value Propositions
Segmentation
PiggybackStudies
Messaging
Competitive Landscape
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Strategic value planning and support
– Integrated market research and outcomes research
– Strategic pricing and positioning
– Strategic economic & outcomes research plans
Retrospective analyses & economic modeling
– Assessments of claims, medical chart, and epidemiology data
– Literature synthesis and meta-analysis
– Burden of illness and budget impact evaluations
– Cost effectiveness and cost-utility analyses
Prospective studies
– Patient registries and observational studies
– Piggyback studies in clinical trials
– Surveys of payers, physicians, patients, others
HRQL / PRO assessment
– Instrument development and validation: health-related quality of life, compliance, preference
– Design & analysis in registries, trials and surveys
Publications and presentations
Dossiers – global value and market-specific
Planning
Information
Building
Communication
Value Demonstration and Risk Management
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Development and Commercialization
Launch
Pre-Clinical• Exploratory Research
• Opportunity
Assessment
• KOL research
• Unmet Needs / Gap Analysis
Phases 1, 2• Clinical trial piggyback
studies- especially HRQL
• Early economic modeling
• Burden of Illness study
• Product Profile Testing
• Unmet Needs /
Gap Analysis
• Exploratory Pricing
• Threshold pricing analysis
• Initial reimbursement
planning
Phase 3, 3b• Clinical trial piggyback studies
• Refined, market-specific
economic models (COI, CEA
CUA, BIM)
• PRO/HRQL analysis for
(1) registration; (2) reimburse-
ment; and/or (3) promotion
• Product positioning
• Messaging/ Value support
• Pricing (price sensitivity
and optimal price)
• Segmentation
• Scenario Testing
• Risk Assessment
• Launch Strategy Design
• Payor Assessment –
pricing and reimbursement
Phase 4• Effectiveness analysis
• Safety surveillance
• Evidence development
• Prospective observational
studies
• Confirmation of CE/CU
• Satisfaction / Loyalty
- Physicians and Patients
• Market Segmentation
• Strategy Evaluation
• Sales Force Effectiveness
• Re-positioning
• Business Analytics
- Sales, revenue targets
• Messaging and Detail Aid
testing
• Pricing
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II. Understanding the
Burden of Disease
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Case Study: To Increase Market Awareness
Retrospective Database Analysis PLUS in Oncology
The Challenge Company had established evidence in clinical trials that a particular health care intervention preserves bone mineral density in men with prostate cancer.
Need to describe the burden of skeletal related events in patients with cancer to build awareness & market.
Approach Given budget and time constraints, strategy was to use retrospective
Medicare data analysis to describe clinical and economic impact of
hormonal therapies on fractures in prostate cancer.
Deliverables Longitudinal Medicare data analysis, thought-leader collaborations, report, presentations, publications
Outcomes Quantified magnitude of the clinical and economic burden and raised awareness among payer (Medicare) and physicians
Multiple presentations at conferences (AUA, ASCO, MASCC) and publications (Cancer and Journal of Clinical Oncology)
Product value dossier for payers, that consolidates evidence
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III. Projecting the Value of
Interventions
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Strategic Modeling Support for Expensive Blood Safety Product
The Challenge A new blood transfusion safety product appears doomed as it isn’t
cost-effective (CER >$250,000/QALY saved)
• Is it possible to shape perceptions regarding product value?
Approach Carefully crafted and implemented a publication plan that “set the
stage” and then delivered release of “noteworthy” CEA results
Deliverables Economic model demonstrates product’s cost effectiveness is
comparable to that of other common safety interventions
• Brochure (for early dissemination)
• Publication on economics of transfusion and product’s CE
• Interactive multi-country model
Outcomes Sponsor senior management praises creative communication
strategy that benefits this and other products
Case Study: To Provide Innovative Strategic Support
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IV. Providing Real World Proof
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Hypertension Program Conference Presentations
Hadker N, Greene B, Brown M, et al. Impact of the BP DownShift Program on Blood Pressure Control among Commercial Driver License Employees: a Managed Care Perspective, Academy of Managed Care Pharmacy 19th Annual Meeting & Showcase, San Diego, CA, April 11-14, 2007.
Greene BL, Brown TM, Harshman RS, et al. Impact of the BP DownShift Program on Blood Pressure Control among Commercial Driver License Employees with Diabetes and Obesity, American Diabetes Association 67th Scientific Sessions, Chicago, June 22-26, 2007.
Greene BL, Brown TM, Harshman RS, et al. Impact of the Change in Department of Transportation (DOT) Guidelines and the BP DownShift Program on Blood Pressure (BP) Control Among Commercial Driver License (CDL) Employees, American Occupational Health Conference, New Orleans, May 6-9, 2007.
Greene BL, Brown TM, Miller JD, Harshman R, Richerson G, Doyle JJ, Economic Impact of the BP DownShift Program on Blood Pressure Control among Commercial Driver License Employees, Academy of Managed Care Pharmacy Educational Conference, Kansas City, Missouri, October 15-18, 2008.
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Hypertension Program Publications
Boolell M, Glasspool J; Cardiovascular Disease Health Burden, Treatment Benefits and Challenges: Need for Partnership, Health & Productivity Management, 2006 (Dec): 5 (4): 11-14.
Foster T, Maintaining a healthy blood pressure among commercial drivers in a utility fleet, Electric Light & Power; 2007 (Sept-Oct); 85 (5): 42-43.
Harshman RS, Richerson GT, Hadker N, et al. Impact of a Hypertension Management/ Health Promotion Program on Commercial Driver’s License Employees of a Self-Insured Utility Company, Journal of Occupational and Environmental Medicine; 2008 (March); 50 (3): 359-365.
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Publication in Journal of Occupational
and Environmental Medicine
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Article in Electric Light & Power
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Conclusions
• Health economics and outcomes research (HEOR) provide
empirical evidence that is complementary to that obtained
from randomized clinical trials
• HEOR publications create awareness of disease burden
and product value among key stakeholders
• HEOR publications are critical for technology registration,
reimbursement and promotion
Questions & Answers
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query into the ‘Q&A’ chat box at the
bottom left of your screen. Every attempt
will be made to answer all questions.
Next ISMPP U
DATE: December 3rd
TIME: 11am EST
TOPIC: Journal Club: The New GPP2 Guidelines
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